Niemann-Pick disease is a rare genetic medical condition. There are four variants of this disease, categorized as type A, type B, type C, and type D. Niemann-Pick disease causes a variety of ...

It never occurred to the family that both brothers could have the same rare genetic condition. In 2017, neurologists at New York Presbyterian/ Columbia University’s movement clinic diagnosed Daniel ...

IntraBio's Aqneursa (levacetylleucine) has joined Zevra Therapeutics' Miplyffa (arimoclomol) for patients with Niemann-Pick type C (NPC), a rare genetic disease affecting around one in 100,000 ...

Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease ... with Niemann-Pick type C (NPC).

To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1‐800-FDA-1088 or .